The California National Primate Research Center is pleased to announce the 2023-2024 CNPRC Pilot Research Program recipients. Every year the CNPRC awards pilot research grants focusing on nonhuman primate (NHP) models of human disease. This award aims to explore innovative areas of NHP research and generate preliminary data to serve as a basis for submitting new research grant applications (e.g., R01-type applications to the National Institute of Health) and/or subsequent clinical studies.
Novel Strategy to Enable Re-administration of Vectors for Gene Therapies and Vaccines
Principle Investigator: Chengwen Li, M.D./PhD
“Gene therapy with adeno-associated virus (AAV) vector has been successfully applied in numerous clinical trials with 6 AAV based drugs approved by the FDA recently. However, more than 50% of patients have antibodies against AAV and been excluded from this effective treatment. We have developed a biomolecule with high efficiency to block AAV antibodies in mice. In this study, we will study the efficacy and safety of this biomolecule in non-human primates. If successful, this biomolecule could be immediately transited into the clinical applications to benefit more patients receiving AAV gene therapy.”
Evaluating the Safety and Efficacy of CD47-IL6 Blockage by Antibody for Reversing Pulmonary Fibrosis in Interstitial Lung Diseases in a Non-Human Primate Model
Principle Investigator: Gerlinde Wernig, M.D.
“Pulmonary fibrosis (PF) and architectural remodeling of tissues disrupt lung function with often fatal consequences. Currently, there are no successful treatments for reversing lung fibrosis. Our previous research revealed effective anti-fibrotic therapeutic strategies to obtain reversion of interstitial lung disease in long COVID patients with PF. Our anti-fibrotic therapeutic strategy is to resolve lung fibrosis in our pre-clinical models at the transcriptional level with significant therapeutic efficacy and minimal or no toxicity. Our goal is to further our approach in non-human primate models for further evaluation of the toxicity and efficacy of our studies.”
Advancing Gene Therapy for Oculocutaneous Albinism
Principle Investigator: Leah Byrne, PhD
“Oculocutaneous albinism (OCA) is a group of disorders that causes reduced pigmentation in the eyes, hair, and skin, resulting in severe visual impairment. Currently, there are no effective treatments for OCA vision loss, making it a significant unmet clinical need. Gene therapy is a promising approach for treating OCA, but we lack animal models with eyes similar to humans in which to develop and test these therapies. Recently, several adult rhesus macaques at CNPRC were identified as having features similar to humans with OCA, including reduced pigmentation in the hair and eye. In this proposal, we will screen NHPs at CNPRC to identify carriers of OCA-causing mutations. We will also perform preliminary testing of OCA gene therapy in primates. Overall, this research may lead to the development of a potential gene therapy treatment for OCA and improve the quality of life for those affected by this disorder.”
Immunomodulatory Effects of Clostridium Immunis in a Non-Human Primate Model
Principle Investigator: Neeraj Surana
“Numerous human diseases are thought to result from an abnormal immune response against the bacteria that normally live in and on every individual (the microbiota), with the idea that “fixing” this microbial community may treat the disease itself. We recently discovered a new human bacterium, Clostridium immunis, that protects mice against metabolic and inflammatory diseases by regulating the immune system. In this pilot project, we aim to determine the safety and efficacy of C. immunis in rhesus macaques. These studies will provide pivotal pre-clinical findings that will help develop C. immunisinto a clinically-relevant therapeutic.”